Exploring retinal degenerative diseases through CRISPR-based screening

Rui Li ^1,2, Fengming Yang ^1,2, Boling Chu ^1,2

¹Sichuan Provincial Key Laboratory for Human Disease Gene Study and the Center for Medical Genetics, Department of Laboratory Medicine, Sichuan Academy of Medical Sciences and Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu, China.
²School of Medicine, University of Electronic Science and Technology of China, Chengdu, China.
³Sichuan Provincial Key Laboratory for Human Disease Gene Study and the Center for Medical Genetics, Department of Laboratory Medicine, Sichuan Academy of Medical Sciences and Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu, China. jinghu_somed@uestc.edu.cn.
⁴School of Medicine, University of Electronic Science and Technology of China, Chengdu, China. jinghu_somed@uestc.edu.cn.
⁵Research Unit for Blindness Prevention, Chinese Academy of Medical Sciences (2019RU026), Sichuan Academy of Medical Sciences and Sichuan Provincial People's Hospital, Chengdu, Sichuan, China. jinghu_somed@uestc.edu.cn.
⁶Sichuan Provincial Key Laboratory for Human Disease Gene Study and the Center for Medical Genetics, Department of Laboratory Medicine, Sichuan Academy of Medical Sciences and Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu, China. qianhao@uestc.edu.cn.
⁷School of Medicine, University of Electronic Science and Technology of China, Chengdu, China. qianhao@uestc.edu.cn.
⁸Research Unit for Blindness Prevention, Chinese Academy of Medical Sciences (2019RU026), Sichuan Academy of Medical Sciences and Sichuan Provincial People's Hospital, Chengdu, Sichuan, China. qianhao@uestc.edu.cn.

⁰Sichuan Provincial Key Laboratory for Human Disease Gene Study and the Center for Medical Genetics, Department of Laboratory Medicine, Sichuan Academy of Medical Sciences and Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu, China.

Molecular Biology Reports +

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September 30, 2024

View abstract on PubMed

Summary

CRISPR screening offers a powerful new approach for understanding retinal degenerative diseases. This review explores CRISPR tools, screening methods, and their application to advance retinal research and develop novel therapies.

Area Of Science

Genetics and Molecular Biology
Ophthalmology
Biotechnology

Background

The CRISPR/Cas9 system is a revolutionary gene-editing technology with broad applications.
CRISPR screening has advanced fields like cancer biology and drug discovery.
Retinal degenerative diseases urgently require new treatments due to poorly understood mechanisms.

Purpose Of The Study

To provide a comprehensive review of CRISPR tools and screening methodologies.
To explore the potential of CRISPR screening in retinal degenerative disease research.
To guide experimental design and identify research questions for retinal applications.

Main Methods

Review of CRISPR/Cas9 system evolution and current screening platforms.
Discussion of experimental design, including selection strategies and delivery methods for CRISPR components (sgRNAs, Cas proteins).
Exploration of suitable in vitro models for retinal CRISPR screening.

Main Results

CRISPR screening has significant, yet largely untapped, potential in retinal research.
Pioneering studies and experimental guidelines for CRISPR screening in retinal contexts are presented.
Identification of key research questions addressable by CRISPR screening in retinal diseases.

Conclusions

This review highlights the transformative potential of CRISPR screening for retinal degenerative disorders.
It aims to foster innovation in understanding retinal pathophysiology.
The findings are expected to accelerate therapeutic development and improve patient outcomes.

Keywords:

CRISPR Screening CRISPR/Cas9 Gene editing Retinal degenerative diseases